U.S. FDA panel backs BioMarin’s Morquio A Syndrome drug

An experimental drug to treat Morquio A Syndrome, a rare genetic disorder that causes skeletal malformation and a variety of related lung, eye, ear and heart problems, should be approved, an advisory panel to the U.S. Food and Drug Administration concluded on Tuesday. The 21-member panel voted overwhelmingly in favor of approval, saying the benefits of the drug, Vimizim, which is made by BioMarin Pharmaceutical Inc, outweigh its risks. The FDA is not obliged to follow the recommendations of its advisory panels but typically does so. Morquio A Syndrome is characterized by a deficiency of an enzyme known as N-acetylgalactosamine-6-sulfatase, which causes excessive storage in the body of long chains of sugars known as glycosaminoglycans.